JP6756700B2 - 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 - Google Patents
筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 Download PDFInfo
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- JP6756700B2 JP6756700B2 JP2017501101A JP2017501101A JP6756700B2 JP 6756700 B2 JP6756700 B2 JP 6756700B2 JP 2017501101 A JP2017501101 A JP 2017501101A JP 2017501101 A JP2017501101 A JP 2017501101A JP 6756700 B2 JP6756700 B2 JP 6756700B2
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
- C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
- C12Y115/01001—Superoxide dismutase (1.15.1.1)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Physical Education & Sports Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Pain & Pain Management (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Neurology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461955189P | 2014-03-18 | 2014-03-18 | |
| US61/955,189 | 2014-03-18 | ||
| PCT/US2015/021321 WO2015143078A1 (en) | 2014-03-18 | 2015-03-18 | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019159514A Division JP7051118B2 (ja) | 2014-03-18 | 2019-09-02 | 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017510298A JP2017510298A (ja) | 2017-04-13 |
| JP2017510298A5 JP2017510298A5 (enExample) | 2018-04-26 |
| JP6756700B2 true JP6756700B2 (ja) | 2020-09-16 |
Family
ID=54145287
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017501101A Active JP6756700B2 (ja) | 2014-03-18 | 2015-03-18 | 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 |
| JP2019159514A Active JP7051118B2 (ja) | 2014-03-18 | 2019-09-02 | 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 |
| JP2021093003A Pending JP2021152030A (ja) | 2014-03-18 | 2021-06-02 | 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 |
| JP2024000089A Pending JP2024032730A (ja) | 2014-03-18 | 2024-01-04 | 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019159514A Active JP7051118B2 (ja) | 2014-03-18 | 2019-09-02 | 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 |
| JP2021093003A Pending JP2021152030A (ja) | 2014-03-18 | 2021-06-02 | 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 |
| JP2024000089A Pending JP2024032730A (ja) | 2014-03-18 | 2024-01-04 | 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (6) | US10280418B2 (enExample) |
| EP (3) | EP3750907A3 (enExample) |
| JP (4) | JP6756700B2 (enExample) |
| AU (3) | AU2015231294B2 (enExample) |
| CA (1) | CA2942515A1 (enExample) |
| DK (1) | DK3119797T3 (enExample) |
| ES (1) | ES2856403T3 (enExample) |
| WO (1) | WO2015143078A1 (enExample) |
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| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| US9394333B2 (en) | 2008-12-02 | 2016-07-19 | Wave Life Sciences Japan | Method for the synthesis of phosphorus atom modified nucleic acids |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| EP2451461A4 (en) | 2009-07-06 | 2013-05-29 | Ontorii Inc | NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF |
| CA2833912C (en) | 2010-04-23 | 2021-09-21 | University Of Massachusetts | Aav-based treatment of cholesterol-related disorders |
| WO2011133874A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Multicistronic expression constructs |
| JP5963743B2 (ja) | 2010-04-23 | 2016-08-03 | ユニバーシティ オブ マサチューセッツ | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| US9226976B2 (en) | 2011-04-21 | 2016-01-05 | University Of Massachusetts | RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
| BR112014001244A2 (pt) | 2011-07-19 | 2017-02-21 | Wave Life Sciences Pte Ltd | métodos para a síntese de ácidos nucléicos funcionalizados |
| CN104684893B (zh) | 2012-07-13 | 2016-10-26 | 日本波涛生命科学公司 | 不对称辅助基团 |
| RU2016133035A (ru) | 2014-01-16 | 2018-02-21 | Уэйв Лайф Сайенсес Лтд. | Хиральный дизайн |
| US10072251B2 (en) | 2014-02-19 | 2018-09-11 | University Of Massachusetts | Recombinant AAVS having useful transcytosis properties |
| US10280418B2 (en) | 2014-03-18 | 2019-05-07 | Univeristy Of Massachusetts | RAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
| WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| US10689653B2 (en) | 2014-06-03 | 2020-06-23 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
| WO2016065001A1 (en) | 2014-10-21 | 2016-04-28 | University Of Massachusetts | Recombinant aav variants and uses thereof |
| CN114717264A (zh) | 2014-11-14 | 2022-07-08 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
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| WO2016172008A1 (en) | 2015-04-24 | 2016-10-27 | University Of Massachusetts | Modified aav constructions and uses thereof |
| US10285388B2 (en) | 2015-05-29 | 2019-05-14 | Regeneron Pharmaceuticals, Inc. | Non-human animals having a disruption in a C9ORF72 locus |
| DK3364997T5 (da) | 2015-10-22 | 2024-09-30 | Univ Massachusetts | Aspartoacylase genterapi til behandling af canavans sygdom |
| EP3364996B1 (en) | 2015-10-22 | 2021-08-25 | University of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
| EP3411059A4 (en) | 2016-02-02 | 2019-10-16 | University Of Massachusetts | METHOD FOR INCREASING THE EFFECTIVENESS OF THE SYSTEMIC DELIVERY OF AVV GENES TO THE CENTRAL NERVOUS SYSTEM |
| US11060088B2 (en) | 2016-02-12 | 2021-07-13 | University Of Massachusetts | Anti-angiogenic miRNA therapeutics for inhibiting corneal neovascularization |
| EP3432933B1 (en) | 2016-03-22 | 2022-10-19 | President and Fellows of Harvard College | Biocompatible adhesives and methods of use thereof |
| EP3436078B1 (en) * | 2016-03-31 | 2021-10-27 | University of Cincinnati | Methods and compositions for the treatment of als |
| CN109714954A (zh) | 2016-04-02 | 2019-05-03 | 全国儿童医院研究所 | 用于组织特异性表达的修饰的u6启动子系统 |
| EP3440210A4 (en) | 2016-04-05 | 2019-11-27 | University of Massachusetts | COMPOSITIONS AND METHOD FOR SELECTIVELY INHIBITING THE EXPRESSION OF GRAINYHEAD LIKE PROTEIN |
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| CN109862785B (zh) * | 2016-09-30 | 2022-09-06 | 瑞泽恩制药公司 | C9orf72基因座中具有六核苷酸重复扩增的非人类动物 |
| AU2017341849B2 (en) | 2016-10-13 | 2024-03-21 | University Of Massachusetts | AAV capsid designs |
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